Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

Phase 2

Terminated

• Conditions: Fragile X Syndrome
• Interventions: Drug: AFQ056

• Registration Number: NCT01348087
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have participated in the previous proof-of-concept study CAFQ056A2204 (NCT00718341).

Detailed Description

A 148 patients were enrolled into this treatment extension trial conducted for at least 3 years. This extension trial was terminated after the decision to terminate the AFQ056 development program. The decision was based on the results of two randomized, double blind, placebo controlled phase IIb trials in adult and adolescent FXS patients (CAFQ056A2212 and CAFQ056B2214respectivly), both of which failed to demonstrate efficacy in the FXS population. As this extension trial was terminated, only the primary objective and safety are represented.

Study Timeline

• Study First Submitted: 2011-05-03
• Study First Submitted QC: 2011-05-04
• Study First Posted: 2011-05-05
• Study Start: 2011-08
• Primary Completion: 2014-09
• Study Completion: 2014-09
• Results First Submitted: 2015-09-09
• Status Verified: 2016-04
• Results First Submitted QC: 2016-04-11
• Last Update Submitted: 2016-04-11
• Results First Posted: 2016-05-25
• Last Update Posted: 2016-05-25

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 148

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AFQ056 100 mg (Bid)	AFQ056	All patients initiated treatment with AFQ056 at a starting dose of 25 milligram (mg) twice daily. The dose was titrated from 25mg bid to 50mg bid, 75mg bid and 100mg bid at weekly intervals. Dose adjustments (up- and down titrations) were permitted as needed to manage any tolerability issues and to ensure that patients reach their highest tolerated dose not to exceed 100mg bid.

Primary Outcome Measures

Name	Time	Method
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).	Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial	Adverse events were summarized for the open-label treatment period, where the open-label treatment period is defined based on how AEs were collected and reported according to the manner in which patients entered the current study and which treatment (AFQ056 or placebo) they were receiving in the previous study. AEs which were continuing from the core study or that started after the end of core study but prior to first dose of open-label study medication in the extension study for Category 1 patients are shown under ('Prior to Ext. first dose'). AEs which started during the open-label treatment period are presented based on the last AFQ056 dose taken on or before the onset date of the AE (25 mg bid; 50 mg bid; 75 mg bid; or 100 mg bid). No efficacy data presented as study was terminated

Trial Locations

Locations (1)

Novartis Investigative Site; 🇬🇧 Edinburgh, United Kingdom